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Patients with genetic lysosomal storage diseases – particularly children – are living longer because of better treatments. But with promising advances and longer lives comes complications, the loss of eyesight as these rare diseases take their toll over time.
Lysosomal storage diseases are inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies, according to the National Organization for Rare Disorders. With these diseases, patients are missing the mucopolysaccharidosis type 1 (MPS1) gene. Current therapies that allow patients to live longer don’t address corneal clouding caused by these diseases. However, one Carolina-affiliated startup, RainBio, is developing a novel gene therapy for MPSI corneal blindness, giving patients a chance not only for longer lives but better ones.
Patients with genetic lysosomal storage diseases – particularly children – are living longer because of better treatments. But with promising advances and longer lives comes complications, the loss of eyesight as these rare diseases take their toll over time.
Lysosomal storage diseases are inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies, according to the National Organization for Rare Disorders. With these diseases, patients are missing the mucopolysaccharidosis type 1 (MPS1) gene. Current therapies that allow patients to live longer don’t address corneal clouding caused by these diseases. However, one Carolina-affiliated startup, RainBio, is developing a novel gene therapy for MPSI corneal blindness, giving patients a chance not only for longer lives but better ones.
rsmith@tribtoday.com
Staff photo / R. Michael Semple
Warren firefighter Brian Gilger, center, receives the inaugural Trumbull County Firefighter of the Year award Friday morning at the Parkman Road fire station, where Gilger is stationed. At left is Jim Standohar, marketing director for Servpro, and at right is Ted Snyder, general manager for the Tribune Chronicle. The newspaper and Servpro co-sponsored the award, which is planned to be an annual one.
WARREN Brian Gilger has been around firefighters all his life.
The 25 1/2 years he has worked for the Warren Fire Department are really the second half of his experience because his father, Dave Gilger, worked for the department for 30 years. The elder Gilger during his last three years of service was able to work side-by-side with his son in the department.